Encouraging Results In Preclinical Studies Of Parkinson's Disease Gene Therapy

Foster City, Calif and Seattle, Wash., January 28, 1999 -- Cell Genesys, Inc. and the University of Washington today published data demonstrating that after a single gene therapy injection, genetically modified mice exhibiting certain symptoms of Parkinson's disease could survive without daily L-dopa treatments for at least one year, the duration of the study. Without L-dopa treatment, these mice die of malnutrition by three weeks of age. An adeno-associated viral (AAV) gene delivery system was used to deliver the genes required for the production of L-dopa to specific regions of the brain where L-dopa production could be detected throughout the observation period. L-dopa is a commonly prescribed drug which is converted to dopamine, the neurotransmitter chemical missing in these mice and in patients with Parkinson's disease. This work was published in the journal, Neuron, by a team of scientists led by Mark Szczypka, Ph.D. and Richard Palmiter, Ph.D. at the University of Washington and Ronald J. Mandel, Ph.D. and Richard O. Snyder, Ph.D. of Cell Genesys.

"A single gene therapy treatment for Parkinson's disease would be a significant improvement over the currently available treatment for this disease," stated Mitchell H. Finer, Ph.D., vice president, research at Cell Genesys. "These studies are among the first observation that gene therapy with AAV vectors can be used to correct a genetic defect in specific regions of the brain."

"The most striking finding in our experiments was the elimination of the need for daily L-dopa treatment in the mice receiving the gene therapy. This represents a remarkable rescue of an otherwise lethal mutation in these genetically modified mice," stated Dr. Richard Palmiter, Investigator of the Howard Hughes Medical Institute and Professor of Biochemistry at the University of Washington. "In the published work, the motivation of the mice to eat and drink was restored following the single administration of

Contact: Laurie McHale
University of Washington

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